Type of information: Granting of a Market Authorisation in the US
Product name: Radicava®
Therapeutic area: Rare diseases - Neurodegenerative diseases
Action mechanism: neuroprotective agent/free radical scavenger
Company: Mitsubishi Tanabe Pharma (Japan)
Disease: amyotrophic lateral sclerosis
- • On May 5, 2017, the FDA approved Radicava® (edaravone) to treat patients with amyotrophic lateral sclerosis. This rare and progressive disease attacks and kills the nerve cells that control voluntary muscles. The nerves lose the ability to activate specific muscles, which causes the muscles to become weak and leads to paralysis. Most people with amyotrophic lateral sclerosi die from respiratory failure, usually within three to five years from when the symptoms first appear. The Centers for Disease Control and Prevention estimates that approximately 12,000-15,000 Americans have amyotrophic lateral sclerosis.
- Radicava® is an intravenous infusion given by a health care professional. It is administered with an initial treatment cycle of daily dosing for 14 days, followed by a 14-day drug-free period. Subsequent treatment cycles consist of dosing on 10 of 14 days, followed by 14 days drug-free.
- The efficacy of edaravone for the treatment of amyotrophic lateral sclerosis was demonstrated in a six-month clinical trial conducted in Japan. In the trial, 137 participants were randomized to receive edaravone or placebo. At Week 24, individuals receiving edaravone declined less on a clinical assessment of daily functioning compared to those receiving a placebo.
- The most common adverse reactions reported by clinical trial participants receiving edaravone were bruising (contusion) and gait disturbance. Radicava® is also associated with serious risks that require immediate medical care, such as hives, swelling, or shortness of breath, and allergic reactions to sodium bisulfite, an ingredient in the drug. Sodium bisulfite may cause anaphylactic symptoms that can be life-threatening in people with sulfite sensitivity.
- • On August 30, 2016, Mitsubishi Tanabe Pharma announced that the FDA has accepted its New Drug Application (NDA) for edavarone (MCI-186) for the treatment of amyotrophic lateral sclerosis. A decision on the application is anticipated on June 16, 2017 based on the Prescription Drug User Fee Fact.
- • On June 20, 2016, Mitsubishi Tanabe Pharma announced that a New Drug Application (NDA) has been submitted to the FDA for edavarone (MCI-186) for the treatment of amyotrophic lateral sclerosis. A decision on the application is anticipated on June 16, 2017 based on the Prescription Drug User Fee Fact.
Submission of marketing authorization application USA : 2016-06-20
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization: 2017-05-05
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA:
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE: